Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

Completed · Phase 2

Conditions studied: Oculopharyngeal Muscular Dystrophy

In brief

The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.

Key facts

Study ID

NCT02015481

Run by

Bioblast Pharma Ltd.

People needed

25

Starts

2014-02-01

Expected to finish

2016-04-01

Last updated by the study team

2017-10-17

Who can join

Age: 18 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Males and females
• 18 - 80 years (inclusive) of age
• Clinically and genetically diagnosed as OPMD
• Moderate dysphagia (abnormal drinking test at screening and on the first dosing day, before drug administration)
• Patients who provide written informed consent to participate in the study
• Body Mass Index (BMI) <30 kg/m2

You may not qualify if…

• Diabetes mellitus type 1 or 2
• Other major diseases, e.g.: renal failure (creatinine clearance <60ml/min), liver failure and chronic liver diseases (e.g. hepatitis B or C) , HIV carriers, tuberculosis, SLE, rheumatoid polyarthritis, sarcoidosis, collagenosis
• Uncontrolled heart disease , CHF,
• Other neuromuscular diseases
• Other disorders associated with esophageal dysphagia: e.g. severe gastroesophageal reflux (GERD), esophageal stricture due to mechanical or chemical trauma, infection (e.g. esophageal moniliasis), drug-induced dysphagia (e.g. bisphosphonates), esophageal rings and webs, spastic motility disorders of the esophagus.
• History of malignancy (except non-invasive skin malignancy)
• History of neck irradiation
• Pregnant or currently lactating women
• Obesity (BMI≥ 30) and associated morbidity
• Prior pharyngeal myotomy
• Weight loss of more than 10% in the last 12 months.
• Known hypersensitivity to any ingredients in the injection
• Patient receiving anticoagulant treatment (e.g. warfarin)

Where it is running

• UCLA — Los Angeles, California, United States
• Tahseen Mozaffar — Orange, California, United States
• Montreal Neurological Institute, McGill University — Montreal, Quebec, Canada
• Hadassah medical center — Jerusalem, Israel

Full record on ClinicalTrials.gov

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